CLINICAL TRIAL DATA
ZILBRYSQ delivered statistically significant and sustained improvements in activities of daily living for adults with anti-AChR Ab+ gMG1,2
Rapid and statistically significant improvements at Week 121,3
In the pivotal Phase 3 RAISE trial, ZILBRYSQ delivered a >4-point improvement in the ability to manage activities of daily living at Week 12 for adults with anti-acetylcholine receptor (AChR) antibody positive (Ab+) generalized Myasthenia Gravis (gMG).
Primary endpoint: Change from baseline (CFB) at Week 12 in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score
The most common adverse reactions (reported in at least 10% of patients treated with ZILBRYSQ) were injection site reactions, upper respiratory tract infections, and diarrhea.1
Clinically meaningful was defined as a ≥2-point change in MG-ADL score.3
The safety and efficacy of ZILBRYSQ were evaluated in a 12-week, multicenter, randomized, double-blind, placebo-controlled study. Patient population included patients with diagnosis of mild to severe gMG (MGFA class II-IV).1
Myasthenia gravis activities of daily living (MG-ADL)
The MG-ADL assesses the impact of gMG on daily functions of 8 symptoms on a scale of 0-3, with total scores ranging from 0 to 24. Higher scores are interpreted as greater impairments.1 An improvement of ≥2 points was established as clinically meaningful.3
Measures include4:
- Talking
- Chewing
- Swallowing
- Breathing
- Brushing teeth and/or combing hair
- Rising from a chair
- Diplopia
- Eyelid droop
Sustained efficacy through Week E482
The primary endpoint of RAISE-XT evaluated the long-term safety and tolerability of ZILBRYSQ at Week E12. Please see the results here.
Includes patients from Phases 2 and 3 of the clinical study who either continued on ZILBRYSQ or switched to ZILBRYSQ from placebo.2
MG-ADL responder rates for ZILBRYSQ in RAISE and RAISE-XT1,2
A high proportion of patients taking ZILBRYSQ were MG-ADL clinical responders (≥ 3-point improvement from baseline) at Week 12 and Week E48.1,2
MG-ADL responder rates in RAISE was an other secondary efficacy endpoint and an exploratory endpoint in RAISE-XT. Results should be interpreted with caution.2,3
Includes patients from Phases 2 and 3 of the clinical study who continued to take ZILBRYSQ in RAISE-XT.2
ZILBRYSQ showed continued MSE in patients through Week E482
Patients who achieved minimal symptom expression (MSE) in either RAISE or RAISE-XT achieved an MG-ADL score of 0 or 1 without rescue therapy.
Patients taking ZILBRYSQ who achieved MSE (mITT population)
MSE was defined as an MG-ADL total score of 0-1. MSE was specified as an other secondary efficacy endpoint in the RAISE study and an exploratory endpoint in RAISE-XT. Results should be interpreted with caution.2,3
Includes patients from Phases 2 and 3 of the clinical study who either continued or switched to ZILBRYSQ from placebo.2
100% of patients who completed the RAISE study opted into RAISE-XT.2
100% of patients who completed the RAISE study opted into RAISE-XT.2
RAISE: consistent and significant improvements across key clinician- and patient-reported secondary outcome measures1,3
Change from baseline at Week 12 in the following endpoints:
Statistically significant improvement in muscle strength1,3
Secondary endpoint: CFB in QMG score at Week 12
The safety and efficacy of ZILBRYSQ were evaluated in a 12-week, multicenter, randomized, double-blind, placebo-controlled study. Patient population included patients with diagnosis of mild to severe gMG (MGFA class II-IV).1
Clinically meaningful was defined as a ≥3-point change in QMG score.3
Statistically significant improvement in gMG signs and symptoms3
Secondary endpoint: CFB in MGC score at Week 12
The safety and efficacy of ZILBRYSQ were evaluated in a 12-week, multicenter, randomized, double-blind, placebo-controlled study. Patient population included patients with diagnosis of mild to severe gMG (MGFA class II-IV).1
Clinically meaningful was defined as a 3-point change in MGC score.3
Statistically significant improvements in quality of life3
Secondary endpoint: CFB in MG-QoL 15r score at Week 12
The safety and efficacy of ZILBRYSQ were evaluated in a 12-week, multicenter, randomized, double-blind, placebo-controlled study. Patient population included patients with diagnosis of mild to severe gMG (MGFA class II-IV).1
Study Limitation: At the time of the study, no threshold for clinical meaningfulness for the MG-QoL 15r assessment had been established.3
Secondary endpoint measures overview
Quantitative Myasthenia Gravis (QMG)
QMG is a physician assessment scoring system that quantifies disease severity based on impairments of body functions and structures. Measures are assessed on a scale of 0-3, with total scores ranging from 0 to 39.1 An improvement of ≥3 points was established as clinically meaningful.3 Measures include6:
- Ptosis
- Facial muscle weakness
- Dysarthria
- Grip strength
- Neck flexion endurance
- Diplopia
- Difficulty swallowing 4 oz of water
- Percentage predicted forced vital capacity
- Arm and leg endurance
Myasthenia Gravis Composite (MGC)
MGC is a 10-item patient and physician assessment of the signs and symptoms of myasthenia gravis based on exam and patient history, with total scores ranging from 0 to 50. Higher scores are interpreted as greater impairments.7 An improvement of ≥3 points was established as clinically meaningful.3 Measures include7:
- Ptosis
- Eye closure
- Chewing
- Breathing
- Shoulder abduction
- Diplopia
- Talking
- Swallowing
- Neck flexion/extension
- Hip flexion
Myasthenia Gravis Quality of Life 15-item revised (MG-QoL 15r) scale
MG-QoL 15r is a 15-item questionnaire that allows clinicians to estimate a patient’s quality of life relevant to MG. Items on the MG-QoL 15r relate to physical, social, and psychological components and are scored from 0 (not at all) to 2 (very much). The cumulative scores range from 0 to 30, with higher scores representing worse quality of life.8 No threshold for clinical meaningfulness for the MG-QoL 15r change had been established.3 Measures include8:
- Frustration
- Trouble eating
- Limitations at work
- Hobby and activity enjoyment
- Depression
- Mobility
- Personal grooming
- Loss of independence
References:
- ZILBRYSQ [Prescribing Information]. Smyrna, GA: UCB, Inc.
- Data on file. UCB, Inc.
- Howard JF Jr, Bresch S, Genge A, et al; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, Phase 3 study. Lancet Neurol. 2023;22(5):395-406. doi:10.1016/S1474-4422(23)00080-7
- MG Activities of Daily Living (MG-ADL) scale. Conquer MG. September 29, 2022. Accessed November 9, 2023. https://www.myastheniagravis.org/mgactivities-of-daily-living-mg-adl-scale/
- Vu T, Genge A, Hussain Y, et al; on behalf of the RAISE investigators. Efficacy and safety of zilucoplan in myasthenia gravis: responder analysis from the randomized Phase 3 RAISE trial: poster 200. Poster presented at: American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting; September 21-24, 2022; Nashville, TN.
- QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed November 9, 2023. https://myasthenia.org/Portals/0/QMG.pdf
- Sadjadi R, Conaway M, Cutter G, et al; MG Composite MG-QOL15 Study Group. Psychometric evaluation of the myasthenia gravis composite using Rasch analysis. Muscle Nerve. 2012;45(6):820-825.
- MG-QOL15R scale. Myasthenia Gravis Rare Disease Network. 2022. Accessed November 9, 2023. https://mgnet.rarediseasesnetwork.org/sites/default/files/2023-04/mg-quality-life-15-revised.pdf
